Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?

Author information

  1. Amsterdam UMC, Clinical Genetics and Amsterdam Public Health Research Institute, Section Community Genetics, Vrije Universiteit Amsterdam, De Boelelaan 1117, Amsterdam, The Netherlands. mc.cornel@vumc.nl.
  2. Centre for Research Ethics and Bioethics, Uppsala University, Box 564, Uppsala, 751 22, Sweden.
  3. Kirkland & Ellis International LLP, London, UK.
  4. Social and Behavioral Research Branch National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  5. Helsinki University Hospital, Clinical Genetics, University of Helsinki, Meilahdentie 2, Helsinki, 00290, Finland.

Abstract

Clinical trials using somatic gene editing (e.g., CRISPR-Cas9) have started in Europe and the United States and may provide safe and effective treatment and cure, not only for cancers but also for some monogenic conditions. In a workshop at the 2018 European Human Genetics Conference, the challenges of bringing somatic gene editing therapies to the clinic were discussed. The regulatory process needs to be considered early in the clinical development pathway to produce the data necessary to support the approval by the European Medicines Agency. The roles and responsibilities for geneticists may include counselling to explain the treatment possibilities and safety interpretation.

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